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    GENE:

    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)

    i
    3d
    Integrated bioinformatics approach for the identification and validation of novel biomarkers in ACC progression and prognosis. (PubMed, Biomarkers)
    Finally, gene-disease and gene-drug association, highlighted correlated diseases and their promising inhibitors. In conclusion, the identified novel biomarkers and associated pathways, provides a comprehensive insight into the molecular mechanisms, prognosis, and potential clinical applications for the diagnosis and therapeutic interventions of ACC.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CXCL12 (C-X-C Motif Chemokine Ligand 12) • IGF1 (Insulin-like growth factor 1) • MMP2 (Matrix metallopeptidase 2) • TAGLN (Transgelin)
    5d
    Oligodendroglial precursor cells modulate immune response and early demyelination in a murine model of multiple sclerosis. (PubMed, Sci Transl Med)
    Antagonizing chemokine receptor 5 (CCR5) using a European Medicines Agency-approved drug, maraviroc, reduced immune cell infiltration, alleviated demyelination, and attenuated EAE progression. We found an OPC-orchestrated immune cellular network that instigates early demyelination, provides insight into MS pathophysiology, and suggests avenues for early interventions.
    Preclinical • Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CD4 (CD4 Molecule) • ITGAM (Integrin, alpha M) • WIF1 (WNT Inhibitory Factor 1) • OLIG2 (Oligodendrocyte Transcription Factor 2)
    |
    Selzentry (maraviroc)
    7d
    Paroxetine ameliorates corticosterone-induced myelin damage by promoting the proliferation and differentiation of oligodendrocyte precursor cells. (PubMed, Neuroscience)
    In vitro, paroxetine significantly increased the viability of primary OPCs and promoted their proliferation and differentiation, with the most potent effect observed at 20 nM. These findings suggest that paroxetine alleviates CORT-induced myelin damage and improves depressive-like behaviors by promoting OPC proliferation and differentiation, providing new insights into its antidepressant mechanisms.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NEFL (Neurofilament Light Chain) • OLIG2 (Oligodendrocyte Transcription Factor 2)
    8d
    Identifying and managing rare subtypes of gastrointestinal stromal tumors. (PubMed, Expert Rev Gastroenterol Hepatol)
    This is a rare and heterogeneous group of challenging GISTs due to their diversity and absence of sensitivity to the tyrosine kinase inhibitor (TKI) imatinib...In SDH-deficient GISTs, the TKI olverembatinib has shown encouraging clinical activity but requires further clinical validation, while the HIF2a inhibitor bezultifan and temozolomide alone or in combination with the death receptor agonist 5 are under clinical investigation...Rare molecular alterations in quadruple WT GISTs require investigation for their oncogenic potential. Collaborative research and patient advocacy is critical for these extremely rare tumors.
    Review • Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • EPAS1 (Endothelial PAS domain protein 1)
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    KIT mutation • RAS mutation • PDGFRA mutation
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    imatinib • temozolomide • Nailike (olverembatinib)
    9d
    Platelet-derived Growth Factor Receptor-α as a Biomarker for Tongue Spindle Cell Carcinoma. (PubMed, Anticancer Res)
    MSC expansion was observed in cancer areas containing EpCAM-positive cells, implying that MSCs, especially PDGFRα-positive MSCs, may serve as cancer niche elements involved in epithelial-mesenchymal transition. Additional studies focusing on PDGFRα pharmacological targeting in SpCC will contribute to understanding of this tumor biology and developing new strategies for treating SpCC.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • EPCAM (Epithelial cell adhesion molecule)
    9d
    CRE25-047: Avapritinib Induced Pseudoprogression in a Gastrointestinal Stromal Tumor With a PDGFRA Exam 18 842 Mutation. (PubMed, J Natl Compr Canc Netw)
    No abstract available
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    PDGFRA mutation
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    Ayvakit (avapritinib)
    10d
    AT101 Suppresses Gastrointestinal Stromal Tumor Growth and Promotes Apoptosis via YAP/TAZ-CCND1 and FBXW7-MCL1 Axes. (PubMed, Ann Surg Oncol)
    AT101 exerts a strong antitumor activity by targeting both the FBXW7-MCL1 axis and the YAP/TAZ-CCND1 pathway, suggesting that AT101 monotherapy, and its combination with IM, are worth further investigating in clinical trials.
    Journal • IO biomarker
    |
    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CCND1 (Cyclin D1) • MCL1 (Myeloid cell leukemia 1) • FBXW7 (F-Box And WD Repeat Domain Containing 7)
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    KIT mutation
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    imatinib
    11d
    Nosologic reappraisal of the recently proposed calcified chondroid mesenchymal neoplasm concept in a series of 20 cases. (PubMed, Mod Pathol)
    Using "CCMN" as an entity would entail mixed repercussions in diagnostic practice, and how and whether it should be used in diagnosis requires deliberate discussion. The concept might bring some benefits, but it may cause more confusion because it would substantially restructure the conventional framework by drawing a boundary within the chondroma and conflating the chondroma with chondroid tenosynovial giant cell tumor.
    Journal
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    FGFR2 (Fibroblast growth factor receptor 2) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • MERTK (MER Proto-Oncogene, Tyrosine Kinase)
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    FGFR2 fusion • PDGFRA rearrangement
    11d
    Chronic Eosinophilic Leukemia Positive for FIP1L1-PDGFRa. (PubMed, Cureus)
    Treatment with imatinib and prednisone led to a rapid decrease in the eosinophil count without further damage from eosinophilic infiltration. Our patient has tolerated therapy with occasional lightheadedness that he attributed to external factors, and he continues to remain in remission. This case study aims to contribute to the existing knowledge regarding the discovery and treatment of this disease.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FIP1L1 (Factor Interacting With PAPOLA And CPSF1) • IL13 (Interleukin 13) • IL4 (Interleukin 4) • IL5 (Interleukin 5)
    |
    imatinib • prednisone
    11d
    Therapeutic Challenges and Emerging Strategies for T674I and PTPN11 Mutations in a FIP1L1-PDGFRA-Positive Myeloproliferative Neoplasm: A Case Report. (PubMed, Life (Basel))
    Myeloproliferative neoplasm (MPN) with eosinophilia associated with FIP1L1-PDGFRA is a rare eosinophilic disorder typically treated with imatinib...Further treatments, including sorafenib and decitabine, failed to control the disease, and the patient ultimately died from multiorgan failure. This case illustrates the therapeutic challenges associated with FIP1L1-PDGFRA T674I-positive eosinophilic disorder, especially when compounded by the PTPN11 mutation. Resistance to standard treatments underscores the urgent need for novel therapies to manage this rare and aggressive disease.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • FIP1L1 (Factor Interacting With PAPOLA And CPSF1)
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    PDGFRA mutation
    |
    sorafenib • imatinib • decitabine
    12d
    Histone demethylase KDM6B promotes glioma cell proliferation by increasing PDGFRA expression via chromatin loop formation. (PubMed, Neurol Res)
    This study reveals a new epigenetic mechanism of PDGFRA overexpression in glioma cells, that is, KDM6B catalyzes the demethylation of H3K27me3 and induces chromatin loop formation to activate PDGFRA expression. This study is of great significance for the understanding of glioma development and the application of new treatment strategies, such as radiation therapy combined with epigenetic therapy.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • KDM6B (Lysine Demethylase 6B)
    12d
    A case of quadruple wild-type gastrointestinal stromal tumor with CDC42BPB::NTRK3 fusion and abundant lymphoid infiltration. (PubMed, Diagn Pathol)
    However, the expression of the Trk protein in tumor tissue was not detected by immunohistochemistry (IHC). This finding expands the genetic spectrum of NTRK rearrangements in GISTs.
    Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • NTRK3 (Neurotrophic tyrosine kinase, receptor, type 3) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NTRK (Neurotrophic receptor tyrosine kinase) • CDC42BPB (CDC42 Binding Protein Kinase Beta)
    |
    PDGFRA mutation
    13d
    Targeting PDGFRA-SHP2 Signaling Enhances Radiotherapy in IDH1-Mutant Glioma. (PubMed, Neuro Oncol)
    Combining SHP2 inhibition with RT is a promising therapeutic avenue for IDH-mut glioma by suppressing the activated SHP2-ERK axis.
    Journal
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    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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    IDH1 mutation • IDH wild-type
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    SHP099
    14d
    Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT. (clinicaltrials.gov)
    P2, N=22, Completed, Sameek Roychowdhury | Active, not recruiting --> Completed
    Trial completion
    |
    FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • RET (Ret Proto-Oncogene) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FGFR (Fibroblast Growth Factor Receptor)
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    FGFR mutation • FGFR fusion
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    Iclusig (ponatinib)
    15d
    Long-term Efficacy and Safety of Benralizumab Treatment for PDGFRA-negative Hypereosinophilic Syndrome. (PubMed, J Allergy Clin Immunol Pract)
    Long-term benralizumab therapy is effective and well-tolerated in HES patients without significant adverse effects.
    Clinical • Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • IL5 (Interleukin 5)
    19d
    Multi-modal magnetic resonance imaging assessment and mechanism exploration of preterm white matter injury in neonatal rats (PubMed, Zhongguo Dang Dai Er Ke Za Zhi)
    Multimodal MRI can evaluate early and long-term changes in PWMI in neonatal rat models in vivo, providing both imaging and pathological evidence for the diagnosis and treatment of PWMI in neonates. Hypoxia-ischemia inhibits the proliferation and differentiation of oligodendrocyte precursor cells in neonatal rats, leading to PWMI.
    Preclinical • Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • OLIG2 (Oligodendrocyte Transcription Factor 2)
    21d
    Cross-family interactions of vascular endothelial growth factors and platelet-derived growth factors on the endothelial cell surface: A computational model. (PubMed, bioRxiv)
    This work provides a foundational computational model for studying cross-family interactions, which can be expanded to investigate tissue-level effects and processes inside cells. Ultimately, our model may help develop better treatments for diseases linked to abnormal blood vessel growth by identifying key protein-receptor interactions.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • KDR (Kinase insert domain receptor) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • FLT1 (Fms-related tyrosine kinase 1) • VEGFB (Vascular Endothelial Growth Factor B) • NRP1 (Neuropilin 1)
    21d
    Characteristics and therapeutic strategies for familial gastrointestinal stromal tumors. (PubMed, World J Gastrointest Oncol)
    The management of GISTs, especially in familial cases, requires a multidisciplinary approach. Cases of different gene mutations were reported in the same family, suggesting that incorporating genetic testing into routine clinical practice is crucial for the early identification of high-risk individuals and the implementation of tailored surveillance programs.
    Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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    KIT mutation • PDGFRA mutation
    21d
    Gastric gastrointestinal stromal tumor in a patient with neurofibromatosis type I presenting with anemia: A case report. (PubMed, World J Gastrointest Oncol)
    Given the diversity of clinical symptoms associated with NF1 and the complexity of NF1-related GISTs, surgical resection with complete tumor removal remains the preferred treatment option. However, the absence of a standardized treatment protocol for adjuvant therapy presents numerous challenges for clinicians.
    Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1) • SDHB (Succinate Dehydrogenase Complex Iron Sulfur Subunit B) • CD34 (CD34 molecule)
    |
    KIT mutation • PDGFRA mutation
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    Stivarga (regorafenib)
    24d
    Effective targeting of PDGFRA-altered high-grade glioma with avapritinib. (PubMed, Cancer Cell)
    Our early data demonstrate that avapritinib is well tolerated and results in radiographic response in 3/7 cases, suggesting a potential role for avapritinib in the treatment of HGG with specific PDGFRA alterations. Overall, these translational results underscore the therapeutic potential of PDGFRA inhibition with avapritinib in HGG.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    PDGFRA mutation
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    Ayvakit (avapritinib)
    26d
    Oligosarcoma with chondroid metaplasia in a French bulldog. (PubMed, J Vet Med Sci)
    In the present case, the tumor had oligodendroglial features accompanied by mesenchymal and chondroid metaplasia. Based on these findings, the dog was diagnosed with an oligosarcoma with chondroid metaplasia.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • VIM (Vimentin) • WWTR1 (WW Domain Containing Transcription Regulator 1) • OLIG2 (Oligodendrocyte Transcription Factor 2)
    26d
    A Cross-Sectional Exploratory Study of Rat Sarcoid (Ras) Activation in Women with and Without Polycystic Ovary Syndrome. (PubMed, Cells)
    After stratifying for BMI ≤ 29.9 kg/m2, EGFR FGF8, FGFR1 VEGF-D, IGF1, and IGF-1sR differed (p < 0.05) though EGF1, EGFRvIII, FGF8, FGFR1, and VEGF-D no longer differed; after subsequently stratifying for HOMA-IR, only FGFR1, VEGF-D, IGF1, and IGF-1sR differed between groups (p < 0.05). Several growth factors that activate Ras differ between women with and without PCOS, and when stratified for BMI and HOMA-IR, only FGFR1, VEGF-D, IGF1, and IGF-1sR differed; these appear to be inherent features of the pathophysiology of PCOS.
    Preclinical • Journal
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    EGFR (Epidermal growth factor receptor) • KRAS (KRAS proto-oncogene GTPase) • NRAS (Neuroblastoma RAS viral oncogene homolog) • FGFR1 (Fibroblast growth factor receptor 1) • HRAS (Harvey rat sarcoma viral oncogene homolog) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • IGF1 (Insulin-like growth factor 1) • VEGFD (Vascular Endothelial Growth Factor D) • FGF8 (Fibroblast Growth Factor 8) • RASA1 (RAS P21 Protein Activator 1)
    28d
    The spectrum of IDH- and H3-wildtype high-grade glioma subgroups occurring across teenage and young adult patient populations. (PubMed, Clin Cancer Res)
    TYA HGGs comprise novel methylation subgroups with distinct molecular profiles. Accurate stratification will open opportunities to utilise more effective treatments.
    Journal
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    EGFR (Epidermal growth factor receptor) • TP53 (Tumor protein P53) • PTEN (Phosphatase and tensin homolog) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • RB1 (RB Transcriptional Corepressor 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1) • MYCN (MYCN Proto-Oncogene BHLH Transcription Factor) • CDK4 (Cyclin-dependent kinase 4) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B) • ATRX (ATRX Chromatin Remodeler) • BCOR (BCL6 Corepressor) • SETD2 (SET Domain Containing 2, Histone Lysine Methyltransferase)
    |
    IDH wild-type
    28d
    Monotherapy vs combination therapy evaluation of FDA-approved TKIs targeting PDGFR for the treatment of thyroid cancer. (PubMed, J Biomol Struct Dyn)
    On 4th May, 2018, a combination of Dabrafenib and Trametinib/Dab-Tra has been approved by US FDA for the treatment of anaplastic thyroid cancer (ATC) that highlights the importance of combination therapy in its treatment...We found that the best combination amongst all is of Pralsetinib and Trametinib/Pra-Tra with a docking score of -14.47 kcal/mol...We also found that a combinatorial approach with TKIs could prove to be better than monotherapy. Further in-vitro and in-vivo studies are required to strengthen the results obtained through in-silico experiment.
    FDA event • Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    Mekinist (trametinib) • Tafinlar (dabrafenib) • Gavreto (pralsetinib)
    29d
    A rare unifocal gastric gastrointestinal stromal tumor in a young NF1 patient: A case report. (PubMed, Int J Surg Case Rep)
    This case highlights the unique clinical and pathological characteristics of NF1-associated GISTs, stressing the need for vigilant assessment in NF1 patients with gastrointestinal symptoms.
    Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1) • CD34 (CD34 molecule) • ANO1 (Anoctamin 1)
    |
    KIT mutation • PDGFRA mutation
    |
    imatinib
    1m
    Selective internal radiation with Y-90 resin microspheres (SIRT) for liver metastases of gastro-intestinal stromal tumors (GIST) resistant to tyrosine kinase inhibitor (TKI) therapy. (PubMed, Br J Cancer)
    90Y-SIRT is a potent treatment for patients with liver metastases of GIST resistant to TKI therapy. In patients with 'q-wt' GIST, SIRT is an option for first-line use.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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    KIT mutation • KIT exon 9 mutation • KIT exon 13 mutation
    1m
    Clinical description and development of a prognostic score for neurofibromatosis type 1 (NF1)-associated GISTs: a retrospective study from the NETSARC. (PubMed, ESMO Open)
    For NF1-GISTs <30 mm, prognosis without relapse is excellent. RECKGIST score accurately predicts recurrence and needs to be validated in an external cohort, but it may help treatment decision making. No efficacy of adjuvant treatment was observed.
    Retrospective data • Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1)
    |
    KIT mutation • PDGFRA mutation
    |
    imatinib
    1m
    Retraction Note: Dynamin 2 mediates PDGFRα-SHP-2-promoted glioblastoma growth and invasion. (PubMed, Oncogene)
    No abstract available
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    1m
    Nuclear translocation of CDK5RAP3 regulated by NXF3 promotes the progression of gastric cancer. (PubMed, Cell Mol Life Sci)
    Our study demonstrates that NXF3 modulates cell cycle progression and promotes gastric cancer development through piRNA-related pathways and the nuclear export of CDK5RAP3 mRNA. Targeting NXF3 represents a promising strategy for developing novel therapeutic approaches for gastric cancer.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CCND1 (Cyclin D1) • IGF1 (Insulin-like growth factor 1) • TGFBR2 (Transforming Growth Factor Beta Receptor 2) • CDKN1A (Cyclin-dependent kinase inhibitor 1A)
    1m
    Impact of Mutation Profile on Outcomes of Neoadjuvant Therapy in GIST. (PubMed, Cancers (Basel))
    Our study highlights the variability in treatment response associated with different GIST mutational profiles. Patients with a KIT-exon-11 mutation tended to respond more favorably to neoadjuvant imatinib in terms of partial response and surgical outcomes.
    Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NTRK (Neurotrophic receptor tyrosine kinase)
    |
    PDGFRA mutation
    |
    imatinib
    1m
    FIP1L1::PDGFRA Fusion in a Pediatric Patient Presenting With B-Cell Lymphoblastic Leukemia. (PubMed, Genes Chromosomes Cancer)
    Identification of FIP1L1::PDGFRA rearrangements is important as they enable treatment with a tyrosine kinase inhibitor, which has significantly improved the overall prognosis for PDGFRA-rearranged neoplasms. Prospective studies assessing imatinib dosage, duration, and long-term safety are warranted in this cohort.
    Journal
    |
    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FIP1L1 (Factor Interacting With PAPOLA And CPSF1)
    |
    FIP1L1-PDGFRA fusion • PDGFRA rearrangement
    |
    imatinib
    1m
    Design, synthesis and pharmacological evaluation of 1,4-naphthoquinone- 1,2,3-triazole hybrids as new anticancer agents with multi-kinase inhibitory activity. (PubMed, Sci Rep)
    Finally, the compounds also possessed drug-like features as examined by in silico studies. The results of this study indicate that 1,4-naphthoquinone derivatives could have promising anticancer potential as multi-kinase inhibitors.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FLT4 (Fms-related tyrosine kinase 4)
    1m
    A Systematic Review with a Demonstrative Case of KIT and DOG-1 Expressing Gastrointestinal Stromal Tumors Harboring ETV6-NTRK3 Fusions. (PubMed, Clin Cancer Res)
    Our findings confirm the existence of ETV6-NTRK3 fusion GIST and demonstrate that these imatinib-resistant GISTs may be exquisitely sensitive to TRK.
    Journal
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • NTRK3 (Neurotrophic tyrosine kinase, receptor, type 3) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • ETV6 (ETS Variant Transcription Factor 6) • NTRK (Neurotrophic receptor tyrosine kinase) • ANO1 (Anoctamin 1)
    |
    KIT expression • NTRK fusion
    |
    Vitrakvi (larotrectinib) • imatinib
    1m
    Clinical Utility of Liquid-based Comprehensive Genomic Profiling (CGP) in Gastrointestinal Stromal Tumors (GIST). (PubMed, Lab Invest)
    55% (42/77) of liquid samples with a KIT-driver mutation had a co-occurring imatinib-resistant alteration; a minority of cases harbored non-KIT mechanisms of resistance such as FGFR2 fusion, BRAF or EGFR alterations...In conclusion, known driver and TKI-resistant mutations were identified in liquid biopsies of GIST patients with high concordance to tissue in the presence of elevated TF. Liquid biopsy may be valuable in the molecular classification and medical management of GIST.
    Journal
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    EGFR (Epidermal growth factor receptor) • BRAF (B-raf proto-oncogene) • FGFR2 (Fibroblast growth factor receptor 2) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1)
    |
    FGFR2 mutation • FGFR2 fusion • KIT exon 13 mutation
    |
    FoundationOne® CDx • FoundationOne® Liquid CDx
    |
    imatinib
    2ms
    Enzymatically responsive nanocarriers targeting PD-1 and TGF-β pathways reverse immunotherapeutic resistance and elicit robust therapeutic efficacy. (PubMed, J Nanobiotechnology)
    We developed gelatinase-responsive nanoparticles (GPNPs) delivering anti-PD-1 antibody (αPD-1) and TGF-β receptor I inhibitor galunisertib (Gal)...Fate mapping demonstrates attenuation of Pdgfrα+ fibroblast transition to αSMA myofibroblasts, potentially reversing "immune-exclusive" status. This study validates GPNPs as a promising lung cancer immunotherapy platform, offering mechanistic insights for clinical translation and therapeutic enhancement.
    Journal • PD(L)-1 Biomarker • IO biomarker
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • TGFB1 (Transforming Growth Factor Beta 1)
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    galunisertib (LY2157299)
    2ms
    Molecular Profiling of Sinonasal Adenoid Cystic Carcinoma: Canonical and Noncanonical Gene Fusions and Mutation. (PubMed, Am J Surg Pathol)
    Noncanonical fusions were predominantly observed in metatypical AdCCs. These findings emphasize the value of comprehensive molecular profiling in correlating morphologic characteristics, genetic landscape, and clinical behavior in AdCC.
    Journal
    |
    PTEN (Phosphatase and tensin homolog) • ROS1 (Proto-Oncogene Tyrosine-Protein Kinase ROS) • RB1 (RB Transcriptional Corepressor 1) • ARID1A (AT-rich interaction domain 1A) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NOTCH1 (Notch 1) • NF1 (Neurofibromin 1) • RUNX1 (RUNX Family Transcription Factor 1) • PALB2 (Partner and localizer of BRCA2) • SMARCA4 (SWI/SNF related, matrix associated, actin dependent regulator of chromatin, subfamily A, member 4) • CREBBP (CREB binding protein) • BCOR (BCL6 Corepressor) • EWSR1 (EWS RNA Binding Protein 1) • KDM6A (Lysine Demethylase 6A) • EP300 (E1A binding protein p300) • DDX41 (DEAD-Box Helicase 41) • PHF6 (PHD Finger Protein 6) • PPM1D (Protein Phosphatase Mg2+/Mn2+ Dependent 1D) • NFIB (Nuclear Factor I B) • RIT1 (Ras Like Without CAAX 1) • CHD2 (Chromodomain Helicase DNA Binding Protein 2) • DNM3 (Dynamin 3) • GEN1 (GEN1 Holliday junction 5' flap endonuclease) • LZTR1 (Leucine Zipper Like Transcription Regulator 1) • MYBL1 (MYB Proto-Oncogene Like 1) • TAF1 (TATA-Box Binding Protein Associated Factor 1)
    2ms
    Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors (clinicaltrials.gov)
    P2, N=50, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Feb 2025 --> Dec 2026 | Trial primary completion date: Feb 2025 --> Dec 2026
    Trial completion date • Trial primary completion date
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    PDGFRA mutation
    |
    FoundationOne® CDx • Guardant360® CDx
    |
    Ayvakit (avapritinib)
    2ms
    AZURE: Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and and Other KIT Altered Hematologic Malignancies (clinicaltrials.gov)
    P1, N=67, Active, not recruiting, Blueprint Medicines Corporation | Recruiting --> Active, not recruiting | Phase classification: P1/2 --> P1 | Trial completion date: Nov 2029 --> Dec 2025 | Trial primary completion date: Nov 2029 --> Jan 2025
    Enrollment closed • Phase classification • Trial completion date • Trial primary completion date
    |
    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    KIT mutation • PDGFRA D842V • PDGFRA mutation
    |
    azacitidine • elenestinib (BLU-263)
    2ms
    PDGFRA::USP8 Fusion in a Cutaneous Inflammatory Myofibroblastic Tumor, Highlighting Genetic Pleiotropy of Kinase Gene Fusions in Soft Tissue Neoplasms. (PubMed, Genes Chromosomes Cancer)
    Herein, we review other soft tissue tumors with PDGFRA fusions, emphasizing PDGFRA::USP8 fusions, further highlighting the genetic pleiotropy of kinase gene fusions in soft tissue tumors. In addition, this case expands the landscape of kinase fusions in IMT, presented by an extremely rare cutaneous IMT.
    Journal
    |
    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    2ms
    A deep-learning model for predicting tyrosine kinase inhibitor response from histology in gastrointestinal stromal tumor. (PubMed, J Pathol)
    To overcome the challenges associated with genetic screening by sequencing, in the current study we developed an artificial intelligence-based deep-learning （DL） model that uses convolutional neural networks (CNN) to analyze digitized hematoxylin and eosin staining in tumor histological sections to predict potential response to imatinib or avapritinib treatment in GIST patients. These results demonstrate the potential of a DL model to improve predictions of treatment response to TKI therapy from histology in GIST patients.
    Journal
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    KIT mutation • PDGFRA mutation
    |
    imatinib • Ayvakit (avapritinib)
    2ms
    NATRON: A Phase III Study to Evaluate the Efficacy and Safety of Benralizumab in Patients With Hypereosinophilic Syndrome (HES) (clinicaltrials.gov)
    P3, N=117, Active, not recruiting, AstraZeneca | Recruiting --> Active, not recruiting | Trial primary completion date: Sep 2024 --> May 2025
    Enrollment closed • Trial primary completion date
    |
    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FIP1L1 (Factor Interacting With PAPOLA And CPSF1)
    |
    FIP1L1-PDGFRA fusion
    2ms
    High-throughput in vitro drug screening and in vivo studies identify fenretinide as a brain-penetrant DMG therapeutic. (PubMed, Neuro Oncol)
    This comprehensive study has identified new DMG therapeutic vulnerabilities and highlights fenretinide as a brain-penetrant, anti-DMG agent.
    Preclinical • Journal
    |
    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)